JP2023534720A5 - - Google Patents

Info

Publication number
JP2023534720A5
JP2023534720A5 JP2023504330A JP2023504330A JP2023534720A5 JP 2023534720 A5 JP2023534720 A5 JP 2023534720A5 JP 2023504330 A JP2023504330 A JP 2023504330A JP 2023504330 A JP2023504330 A JP 2023504330A JP 2023534720 A5 JP2023534720 A5 JP 2023534720A5
Authority
JP
Japan
Prior art keywords
intron
antisense oligonucleotide
sequence
seq
target region
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023504330A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023534720A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/AU2021/050788 external-priority patent/WO2022016222A1/en
Publication of JP2023534720A publication Critical patent/JP2023534720A/ja
Publication of JP2023534720A5 publication Critical patent/JP2023534720A5/ja
Pending legal-status Critical Current

Links

JP2023504330A 2020-07-22 2021-07-22 Scn2a中の機能喪失変異に関連する障害を処置するための組成物及び方法 Pending JP2023534720A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2020902550A AU2020902550A0 (en) 2020-07-22 Compositions and methods for treating disorders associated with loss-of-function mutations in SCN2A
AU2020902550 2020-07-22
PCT/AU2021/050788 WO2022016222A1 (en) 2020-07-22 2021-07-22 Compositions and methods for treating disorders associated with loss-of-function mutations in scn2a

Publications (2)

Publication Number Publication Date
JP2023534720A JP2023534720A (ja) 2023-08-10
JP2023534720A5 true JP2023534720A5 (enExample) 2024-07-18

Family

ID=79729542

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023504330A Pending JP2023534720A (ja) 2020-07-22 2021-07-22 Scn2a中の機能喪失変異に関連する障害を処置するための組成物及び方法

Country Status (15)

Country Link
US (1) US20230272387A1 (enExample)
EP (1) EP4185697A4 (enExample)
JP (1) JP2023534720A (enExample)
KR (1) KR20230095056A (enExample)
CN (1) CN116368227A (enExample)
AU (1) AU2021311137A1 (enExample)
BR (1) BR112023000988A2 (enExample)
CA (1) CA3186629A1 (enExample)
CL (1) CL2023000208A1 (enExample)
CO (1) CO2023001922A2 (enExample)
EC (1) ECSP23012641A (enExample)
IL (1) IL299999A (enExample)
MX (1) MX2023000907A (enExample)
PE (1) PE20230982A1 (enExample)
WO (1) WO2022016222A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4473113A4 (en) * 2022-02-04 2026-03-25 Praxis Prec Medicines Inc TREATMENT METHODS FOR DISORDERS RELATED TO SCN2A
KR20250180156A (ko) 2024-06-21 2025-12-31 중앙대학교 산학협력단 뇌전증의 예방 또는 치료용 조성물, 및 뇌전증 치료제 스크리닝 방법과 이의 조성물

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE1252330T1 (de) * 1999-11-26 2003-11-27 Mcgill University, Montreal Loci der idiopathischen epilepsie, mutationen derselben und verfahren zu deren verwendung zur feststellung, prognose und behandlung von epilepsie
EP1268856A2 (de) * 2000-04-07 2003-01-02 Epigenomics AG Detektion von snp's und cytosin-methylierungen
AU2001276919A1 (en) * 2000-07-13 2002-01-30 Genaissance Pharmaceuticals, Inc. Haplotypes of the mmp13 gene
CA2541836A1 (en) * 2003-10-13 2005-04-21 Bionomics Limited A diagnostic method for neonatal or infantile epilepsy syndromes
CN101148665A (zh) * 2005-12-05 2008-03-26 广州医学院第二附属医院 钠离子通道scn1a基因突变及基因突变检测方法和用途
WO2010151671A2 (en) * 2009-06-24 2010-12-29 Curna, Inc. Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
EP2490699A1 (en) * 2009-10-20 2012-08-29 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
CN109843914B (zh) * 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
KR20190030833A (ko) * 2017-09-15 2019-03-25 가톨릭대학교 산학협력단 신장암 환자의 예후 진단 및 치료 전략 결정용 연령 특이적 마커
HUE070436T2 (hu) * 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
US11713463B2 (en) * 2018-01-17 2023-08-01 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for increasing expression of SCN2A
BR112021003224A2 (pt) * 2018-08-20 2021-07-20 Rogcon, Inc. oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns

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